In 2012, a group of pediatric dermatologists envisioned and created the framework for a collaborative research network, the Pediatric Dermatology Research Alliance (PeDRA). This article explains the origins and progress in creating this novel and exciting work, which has already begun to have an impact on the volume and quality of research studies in the field of pediatric dermatology.
Figure 1. Author Ilona Frieden, MD (second from left) and colleague Beth Drolet, MD (first on right) stand with representatives of thepatient advocacy PHACE Community Syndrome in front of the PHACE poster during the 2014 PeDRA Annual Conference.
Research Needs in Pediatric Dermatology
There are many unmet needs in pediatric dermatology. Many pediatric skin diseases are so uncommon that meaningful study is difficult without collaborative effort. Conducting clinical trials in young children — even in common diseases — is also challenging for many reasons. Recruitment of eligible subjects can be difficult. Most pediatric dermatologists have busy clinical practices and little time, poor funding and lack of infrastructure to do high-quality research. These challenges mean that many — perhaps most — of our therapies for pediatric skin disease are based on anecdotal evidence, expert opinion and precedent. There is a lack of accepted clinical guidelines for many dermatology conditions, including some that are truly life-threatening.
The types of standardized treatment protocols that exist in pediatric oncology and in pediatric rheumatology are sorely lacking in pediatric dermatology, and there is a paucity of National Institutes of Health (NIH)-funded research dollars allotted to pediatric dermatology. Better evidenced-based management for children with skin disorders requires well-designed, multicenter, collaborative clinical trials.
Research Alliance
Members of the Society for Pediatric Dermatology (SPD) have recognized that need to advance research in pediatric dermatology. Historically, there has been no other existing organization specifically dedicated to fostering the growth of research in pediatric dermatologic diseases. Prior to PeDRA’s formation, collaborative research was often informal, though some groups had formed to study important conditions, including infantile hemangiomas, ichthyosis and epidermolysis bullosa. SPD leaders were also aware of the inspiring collaborative work conducted in other pediatric specialties, including pediatric rheumatology and pediatric oncology. These models and a growing interest in collaborative research among pediatric dermatologists helped provide the impetus for the creation of a structure to advance research in pediatric dermatology in order to improve outcomes and quality of life for children with skin diseases.
An inaugural meeting to develop plans for a collaborative clinical and translational pediatric dermatology research network was held in July 2012. Members of the SPD dedicated to clinical research, SPD leadership, NIH staff and other stakeholders attended this meeting and helped to create a roadmap for a new organization to help achieve these goals. Thus, PeDRA was born, supported by multiyear seed money from SPD. PeDRA’s mission is to promote and facilitate high-quality collaborative clinical, translational, educational and basic science research in pediatric dermatology with the vision to create sustainable collaborative research networks to better understand, prevent, treat and cure dermatologic diseases in children.
To this end, PeDRA has developed a leadership structure to drive this work headed by an executive committee comprised of leading researchers and clinicians from around North America. Communications, fundraising, membership and science working committees have also been established. The Alliance established a seminal website, https://pedraresearch.org, to capture pediatric dermatology research priorities, progress on collaborative studies, proceedings of PeDRA annual conferences and other resources relevant to clinicians, researchers and other stakeholders.
“Despite the unparalleled scientific advancements in cutaneous biology and new therapeutics that are emerging, there has been a relative paucity of discovery related to pediatric skin disorders and certainly few translations into new interventions. Importantly, PeDRA has brought together many talented and dedicated individuals who will collectively study rare and common pediatric disorders in a way that we’ve never before been able to pursue at a single site or even through a few collaborating centers." ~Amy Paller, MS, MD, and Lawrence Eichenfield, MD, PeDRA co-chairs
Early Successes
PeDRA leaders recognized the need for a freestanding meeting to create a bridge between basic scientists and clinicians in order to enhance opportunities for translational research initiatives. Successful funding for an annual conference was obtained via a NIH “R13” funding, thus far resulting in 2 stand-alone conferences to identify gaps and challenges in current practice, establish research priorities and develop collaborative clinical, translational and basic science research.
These conferences (held in October 2013 and November 2014) in part involved new investigators, helping them become involved in studies and work alongside seasoned, senior investigators. An important goal is to help trainees and junior clinical pediatric dermatologists develop skills and infrastructure to support their research pursuits. Inclusion of research scientists at these conferences is a key aspect of the meetings, to help advance translational pediatric dermatology research initiatives.
The first freestanding PeDRA research meeting was held in Chicago in October 2013 with more than 80 participants, and was met with great excitement. It focused on teaching key strategies for recruiting and retaining study participants, navigating institutional review boards, writing grant proposals and developing an infrastructure for multicenter collaborative research, with lessons delivered from our pediatric oncology colleagues. Many research proposals were put forth amid much networking.
PeDRA’s second annual conference was held in Chicago in November 2014. More than 110 people attended, including pediatric dermatologists and researchers, pediatric dermatology fellows and dermatology residents, invited speakers and representatives of 7 patient advocacy organizations and of the National Institute of Arthritis and Musculoskeletal and Skin Diseases, a branch of the NIH. There were 39 poster abstracts presented.
During the 3-day meeting, attendees focused on advancing collaborative networks for research to address important problems in pediatric dermatology, guided by a faculty of nationally and internationally recognized speakers. Speakers presented their ongoing research and addressed common issues in collaborative research projects, while breakout sessions provided attendees the opportunity to apply what they learned to new and ongoing projects. In both meetings, pilot and early project ideas were discussed with input from experts and disease advocacy groups.
“PeDRA is living proof that the power of one is absolutely multiplied many fold when people are linked together in collaborative work. After 30 plus years in the field, I feel a new sense of optimism about the future for research in pediatric dermatology.”
~ Moise Levy, MD, PeDRA Executive Committee
PeDRA has focused its initial efforts to foster research and collaboration in several specific disease areas by forming study groups in the following subject areas:
- Birthmarks (vascular & pigment)
- Inflammatory skin disease (including psoriasis, connective tissue & auto-inflammatory, atopic dermatitis, acne and hidradenitis and hair/nail/special sites disorders)
- Genetic skin disorders (including subgroups in epidermolysis bullosa and diseases of cornification)
- Skin tumors and reactions to cancer therapies (including basal cell carcinoma nevus syndrome)
- Neonatal skincare
Additional research groups interested in other topic areas including procedural pediatric dermatology are in the process of being formed.
Significant progress has already been made in each of these areas including identifying funding for specific projects. Studies already launched include a multicenter study of vascular overgrowth syndromes, a morphea clinical registry and biobank, participation in a Yale-sponsored disorders of keratinization project to study a variety of ichthyoses and related disorders and the Epidermolysis Bullosa Clinical Characterization and Outcomes Database.
Two of these research areas have received partial funding from non-traditional sources, spurred on by the inspiring story that PeDRA tells of people joining together to shape and create the research necessary to make a difference for our patients. Morphea is one research area that has been supported. An ichthyosis newborn study has been partially funded in order to track babies born with the disease, record changes over time and correlate to the type of ichthyosis the baby has. This will generate historical data to help dermatologists better recognize and understand the natural course of the disease that associates with different specific types of ichthyosis.
Figure 2. Megha Tollefson, MD, Mayo Clinic and member of the PeDRA network, works with pediatric patient.
Conclusions
By forming a network of researchers, PeDRA has the potential to transform research in pediatric dermatology by multiplying the power of individual scientists, linking them together to enable critical collaboration to address the important gaps in the understanding of skin disorders of childhood. PeDRA is growing rapidly and consists of approximately 150 scientists, clinical investigators and clinicians working together in order to positively impact the lives of children living with dermatologic diseases. However, there is still much more to do. n
Dr. Sugarman is associate clinical professor, University of California, San Francisco and in practice in Santa Rosa, CA.
Dr. Frieden is professor of dermatology and pediatrics, University of California, San Francisco.
In 2012, a group of pediatric dermatologists envisioned and created the framework for a collaborative research network, the Pediatric Dermatology Research Alliance (PeDRA). This article explains the origins and progress in creating this novel and exciting work, which has already begun to have an impact on the volume and quality of research studies in the field of pediatric dermatology.
Figure 1. Author Ilona Frieden, MD (second from left) and colleague Beth Drolet, MD (first on right) stand with representatives of thepatient advocacy PHACE Community Syndrome in front of the PHACE poster during the 2014 PeDRA Annual Conference.
Research Needs in Pediatric Dermatology
There are many unmet needs in pediatric dermatology. Many pediatric skin diseases are so uncommon that meaningful study is difficult without collaborative effort. Conducting clinical trials in young children — even in common diseases — is also challenging for many reasons. Recruitment of eligible subjects can be difficult. Most pediatric dermatologists have busy clinical practices and little time, poor funding and lack of infrastructure to do high-quality research. These challenges mean that many — perhaps most — of our therapies for pediatric skin disease are based on anecdotal evidence, expert opinion and precedent. There is a lack of accepted clinical guidelines for many dermatology conditions, including some that are truly life-threatening.
The types of standardized treatment protocols that exist in pediatric oncology and in pediatric rheumatology are sorely lacking in pediatric dermatology, and there is a paucity of National Institutes of Health (NIH)-funded research dollars allotted to pediatric dermatology. Better evidenced-based management for children with skin disorders requires well-designed, multicenter, collaborative clinical trials.
Research Alliance
Members of the Society for Pediatric Dermatology (SPD) have recognized that need to advance research in pediatric dermatology. Historically, there has been no other existing organization specifically dedicated to fostering the growth of research in pediatric dermatologic diseases. Prior to PeDRA’s formation, collaborative research was often informal, though some groups had formed to study important conditions, including infantile hemangiomas, ichthyosis and epidermolysis bullosa. SPD leaders were also aware of the inspiring collaborative work conducted in other pediatric specialties, including pediatric rheumatology and pediatric oncology. These models and a growing interest in collaborative research among pediatric dermatologists helped provide the impetus for the creation of a structure to advance research in pediatric dermatology in order to improve outcomes and quality of life for children with skin diseases.
An inaugural meeting to develop plans for a collaborative clinical and translational pediatric dermatology research network was held in July 2012. Members of the SPD dedicated to clinical research, SPD leadership, NIH staff and other stakeholders attended this meeting and helped to create a roadmap for a new organization to help achieve these goals. Thus, PeDRA was born, supported by multiyear seed money from SPD. PeDRA’s mission is to promote and facilitate high-quality collaborative clinical, translational, educational and basic science research in pediatric dermatology with the vision to create sustainable collaborative research networks to better understand, prevent, treat and cure dermatologic diseases in children.
To this end, PeDRA has developed a leadership structure to drive this work headed by an executive committee comprised of leading researchers and clinicians from around North America. Communications, fundraising, membership and science working committees have also been established. The Alliance established a seminal website, https://pedraresearch.org, to capture pediatric dermatology research priorities, progress on collaborative studies, proceedings of PeDRA annual conferences and other resources relevant to clinicians, researchers and other stakeholders.
“Despite the unparalleled scientific advancements in cutaneous biology and new therapeutics that are emerging, there has been a relative paucity of discovery related to pediatric skin disorders and certainly few translations into new interventions. Importantly, PeDRA has brought together many talented and dedicated individuals who will collectively study rare and common pediatric disorders in a way that we’ve never before been able to pursue at a single site or even through a few collaborating centers." ~Amy Paller, MS, MD, and Lawrence Eichenfield, MD, PeDRA co-chairs
Early Successes
PeDRA leaders recognized the need for a freestanding meeting to create a bridge between basic scientists and clinicians in order to enhance opportunities for translational research initiatives. Successful funding for an annual conference was obtained via a NIH “R13” funding, thus far resulting in 2 stand-alone conferences to identify gaps and challenges in current practice, establish research priorities and develop collaborative clinical, translational and basic science research.
These conferences (held in October 2013 and November 2014) in part involved new investigators, helping them become involved in studies and work alongside seasoned, senior investigators. An important goal is to help trainees and junior clinical pediatric dermatologists develop skills and infrastructure to support their research pursuits. Inclusion of research scientists at these conferences is a key aspect of the meetings, to help advance translational pediatric dermatology research initiatives.
The first freestanding PeDRA research meeting was held in Chicago in October 2013 with more than 80 participants, and was met with great excitement. It focused on teaching key strategies for recruiting and retaining study participants, navigating institutional review boards, writing grant proposals and developing an infrastructure for multicenter collaborative research, with lessons delivered from our pediatric oncology colleagues. Many research proposals were put forth amid much networking.
PeDRA’s second annual conference was held in Chicago in November 2014. More than 110 people attended, including pediatric dermatologists and researchers, pediatric dermatology fellows and dermatology residents, invited speakers and representatives of 7 patient advocacy organizations and of the National Institute of Arthritis and Musculoskeletal and Skin Diseases, a branch of the NIH. There were 39 poster abstracts presented.
During the 3-day meeting, attendees focused on advancing collaborative networks for research to address important problems in pediatric dermatology, guided by a faculty of nationally and internationally recognized speakers. Speakers presented their ongoing research and addressed common issues in collaborative research projects, while breakout sessions provided attendees the opportunity to apply what they learned to new and ongoing projects. In both meetings, pilot and early project ideas were discussed with input from experts and disease advocacy groups.
“PeDRA is living proof that the power of one is absolutely multiplied many fold when people are linked together in collaborative work. After 30 plus years in the field, I feel a new sense of optimism about the future for research in pediatric dermatology.”
~ Moise Levy, MD, PeDRA Executive Committee
PeDRA has focused its initial efforts to foster research and collaboration in several specific disease areas by forming study groups in the following subject areas:
- Birthmarks (vascular & pigment)
- Inflammatory skin disease (including psoriasis, connective tissue & auto-inflammatory, atopic dermatitis, acne and hidradenitis and hair/nail/special sites disorders)
- Genetic skin disorders (including subgroups in epidermolysis bullosa and diseases of cornification)
- Skin tumors and reactions to cancer therapies (including basal cell carcinoma nevus syndrome)
- Neonatal skincare
Additional research groups interested in other topic areas including procedural pediatric dermatology are in the process of being formed.
Significant progress has already been made in each of these areas including identifying funding for specific projects. Studies already launched include a multicenter study of vascular overgrowth syndromes, a morphea clinical registry and biobank, participation in a Yale-sponsored disorders of keratinization project to study a variety of ichthyoses and related disorders and the Epidermolysis Bullosa Clinical Characterization and Outcomes Database.
Two of these research areas have received partial funding from non-traditional sources, spurred on by the inspiring story that PeDRA tells of people joining together to shape and create the research necessary to make a difference for our patients. Morphea is one research area that has been supported. An ichthyosis newborn study has been partially funded in order to track babies born with the disease, record changes over time and correlate to the type of ichthyosis the baby has. This will generate historical data to help dermatologists better recognize and understand the natural course of the disease that associates with different specific types of ichthyosis.
Figure 2. Megha Tollefson, MD, Mayo Clinic and member of the PeDRA network, works with pediatric patient.
Conclusions
By forming a network of researchers, PeDRA has the potential to transform research in pediatric dermatology by multiplying the power of individual scientists, linking them together to enable critical collaboration to address the important gaps in the understanding of skin disorders of childhood. PeDRA is growing rapidly and consists of approximately 150 scientists, clinical investigators and clinicians working together in order to positively impact the lives of children living with dermatologic diseases. However, there is still much more to do. n
Dr. Sugarman is associate clinical professor, University of California, San Francisco and in practice in Santa Rosa, CA.
Dr. Frieden is professor of dermatology and pediatrics, University of California, San Francisco.
In 2012, a group of pediatric dermatologists envisioned and created the framework for a collaborative research network, the Pediatric Dermatology Research Alliance (PeDRA). This article explains the origins and progress in creating this novel and exciting work, which has already begun to have an impact on the volume and quality of research studies in the field of pediatric dermatology.
Figure 1. Author Ilona Frieden, MD (second from left) and colleague Beth Drolet, MD (first on right) stand with representatives of thepatient advocacy PHACE Community Syndrome in front of the PHACE poster during the 2014 PeDRA Annual Conference.
Research Needs in Pediatric Dermatology
There are many unmet needs in pediatric dermatology. Many pediatric skin diseases are so uncommon that meaningful study is difficult without collaborative effort. Conducting clinical trials in young children — even in common diseases — is also challenging for many reasons. Recruitment of eligible subjects can be difficult. Most pediatric dermatologists have busy clinical practices and little time, poor funding and lack of infrastructure to do high-quality research. These challenges mean that many — perhaps most — of our therapies for pediatric skin disease are based on anecdotal evidence, expert opinion and precedent. There is a lack of accepted clinical guidelines for many dermatology conditions, including some that are truly life-threatening.
The types of standardized treatment protocols that exist in pediatric oncology and in pediatric rheumatology are sorely lacking in pediatric dermatology, and there is a paucity of National Institutes of Health (NIH)-funded research dollars allotted to pediatric dermatology. Better evidenced-based management for children with skin disorders requires well-designed, multicenter, collaborative clinical trials.
Research Alliance
Members of the Society for Pediatric Dermatology (SPD) have recognized that need to advance research in pediatric dermatology. Historically, there has been no other existing organization specifically dedicated to fostering the growth of research in pediatric dermatologic diseases. Prior to PeDRA’s formation, collaborative research was often informal, though some groups had formed to study important conditions, including infantile hemangiomas, ichthyosis and epidermolysis bullosa. SPD leaders were also aware of the inspiring collaborative work conducted in other pediatric specialties, including pediatric rheumatology and pediatric oncology. These models and a growing interest in collaborative research among pediatric dermatologists helped provide the impetus for the creation of a structure to advance research in pediatric dermatology in order to improve outcomes and quality of life for children with skin diseases.
An inaugural meeting to develop plans for a collaborative clinical and translational pediatric dermatology research network was held in July 2012. Members of the SPD dedicated to clinical research, SPD leadership, NIH staff and other stakeholders attended this meeting and helped to create a roadmap for a new organization to help achieve these goals. Thus, PeDRA was born, supported by multiyear seed money from SPD. PeDRA’s mission is to promote and facilitate high-quality collaborative clinical, translational, educational and basic science research in pediatric dermatology with the vision to create sustainable collaborative research networks to better understand, prevent, treat and cure dermatologic diseases in children.
To this end, PeDRA has developed a leadership structure to drive this work headed by an executive committee comprised of leading researchers and clinicians from around North America. Communications, fundraising, membership and science working committees have also been established. The Alliance established a seminal website, https://pedraresearch.org, to capture pediatric dermatology research priorities, progress on collaborative studies, proceedings of PeDRA annual conferences and other resources relevant to clinicians, researchers and other stakeholders.
“Despite the unparalleled scientific advancements in cutaneous biology and new therapeutics that are emerging, there has been a relative paucity of discovery related to pediatric skin disorders and certainly few translations into new interventions. Importantly, PeDRA has brought together many talented and dedicated individuals who will collectively study rare and common pediatric disorders in a way that we’ve never before been able to pursue at a single site or even through a few collaborating centers." ~Amy Paller, MS, MD, and Lawrence Eichenfield, MD, PeDRA co-chairs
Early Successes
PeDRA leaders recognized the need for a freestanding meeting to create a bridge between basic scientists and clinicians in order to enhance opportunities for translational research initiatives. Successful funding for an annual conference was obtained via a NIH “R13” funding, thus far resulting in 2 stand-alone conferences to identify gaps and challenges in current practice, establish research priorities and develop collaborative clinical, translational and basic science research.
These conferences (held in October 2013 and November 2014) in part involved new investigators, helping them become involved in studies and work alongside seasoned, senior investigators. An important goal is to help trainees and junior clinical pediatric dermatologists develop skills and infrastructure to support their research pursuits. Inclusion of research scientists at these conferences is a key aspect of the meetings, to help advance translational pediatric dermatology research initiatives.
The first freestanding PeDRA research meeting was held in Chicago in October 2013 with more than 80 participants, and was met with great excitement. It focused on teaching key strategies for recruiting and retaining study participants, navigating institutional review boards, writing grant proposals and developing an infrastructure for multicenter collaborative research, with lessons delivered from our pediatric oncology colleagues. Many research proposals were put forth amid much networking.
PeDRA’s second annual conference was held in Chicago in November 2014. More than 110 people attended, including pediatric dermatologists and researchers, pediatric dermatology fellows and dermatology residents, invited speakers and representatives of 7 patient advocacy organizations and of the National Institute of Arthritis and Musculoskeletal and Skin Diseases, a branch of the NIH. There were 39 poster abstracts presented.
During the 3-day meeting, attendees focused on advancing collaborative networks for research to address important problems in pediatric dermatology, guided by a faculty of nationally and internationally recognized speakers. Speakers presented their ongoing research and addressed common issues in collaborative research projects, while breakout sessions provided attendees the opportunity to apply what they learned to new and ongoing projects. In both meetings, pilot and early project ideas were discussed with input from experts and disease advocacy groups.
“PeDRA is living proof that the power of one is absolutely multiplied many fold when people are linked together in collaborative work. After 30 plus years in the field, I feel a new sense of optimism about the future for research in pediatric dermatology.”
~ Moise Levy, MD, PeDRA Executive Committee
PeDRA has focused its initial efforts to foster research and collaboration in several specific disease areas by forming study groups in the following subject areas:
- Birthmarks (vascular & pigment)
- Inflammatory skin disease (including psoriasis, connective tissue & auto-inflammatory, atopic dermatitis, acne and hidradenitis and hair/nail/special sites disorders)
- Genetic skin disorders (including subgroups in epidermolysis bullosa and diseases of cornification)
- Skin tumors and reactions to cancer therapies (including basal cell carcinoma nevus syndrome)
- Neonatal skincare
Additional research groups interested in other topic areas including procedural pediatric dermatology are in the process of being formed.
Significant progress has already been made in each of these areas including identifying funding for specific projects. Studies already launched include a multicenter study of vascular overgrowth syndromes, a morphea clinical registry and biobank, participation in a Yale-sponsored disorders of keratinization project to study a variety of ichthyoses and related disorders and the Epidermolysis Bullosa Clinical Characterization and Outcomes Database.
Two of these research areas have received partial funding from non-traditional sources, spurred on by the inspiring story that PeDRA tells of people joining together to shape and create the research necessary to make a difference for our patients. Morphea is one research area that has been supported. An ichthyosis newborn study has been partially funded in order to track babies born with the disease, record changes over time and correlate to the type of ichthyosis the baby has. This will generate historical data to help dermatologists better recognize and understand the natural course of the disease that associates with different specific types of ichthyosis.
Figure 2. Megha Tollefson, MD, Mayo Clinic and member of the PeDRA network, works with pediatric patient.
Conclusions
By forming a network of researchers, PeDRA has the potential to transform research in pediatric dermatology by multiplying the power of individual scientists, linking them together to enable critical collaboration to address the important gaps in the understanding of skin disorders of childhood. PeDRA is growing rapidly and consists of approximately 150 scientists, clinical investigators and clinicians working together in order to positively impact the lives of children living with dermatologic diseases. However, there is still much more to do. n
Dr. Sugarman is associate clinical professor, University of California, San Francisco and in practice in Santa Rosa, CA.
Dr. Frieden is professor of dermatology and pediatrics, University of California, San Francisco.
